POS1258 RESULTS FROM A NOMINAL GROUP TECHNIQUE EXERCISE FOR THE CREATION OF A COMBINED RESPONSE INDEX FOR LIMITED CUTANEOUS SYSTEMIC SCLEROSIS: THE CRISTAL PROJECT
Authors: A. Lescoat, Y. Chen, S. L. Murphy, L. Carroll, N. Vann, P. Didio, S. Iya, T. Phanhdone, S. Farrington, Y. Allanore, D. Cella, L. Chung, P. Clements, F. Del Galdo, C. P. Denton, O. Distler, M. Hinchcliff, M. Hughes, L. Hummers, J. Pauling, J. Pope, V. Steen, J. Varga, M. H. Buch, D. Khanna
Journal: Annals of the Rheumatic Diseases (2023)

<h3>Background</h3> The lack of RCTs in limited cutaneous systemic sclerosis (lcSSc) is due, in part, to a paucity of relevant outcome measures to effectively evaluate this subset. This international project, supported by the World Scleroderma Foundation (WSF), Scleroderma &amp;. Raynaud's UK (SRUK), the Scleroderma Clinical Trial Consortium (SCTC), and the US National Scleroderma Foundation, aims to create a combined response index for lcSSc for use in clinical trials; the CRISTAL index. The most bothersome symptoms from the patients' perspective were identified through lcSSc patient-centered focus groups [1] and already-identified domains and outcomes were identified through a scoping literature review [2]. <h3>Objectives</h3> This phase of the overall CRISTAL project aimed to select candidate items for inclusion in a planned longitudinal prospective observational study assessing the psychometric properties of the selected items. We aimed to enrich the list of candidate outcomes from domains identified by the focus groups and scoping review: i) SSc-specific domains (gastro-intestinal involvement, Raynaud's/microcirculation, musculoskeletal, digital ulcers, pulmonary involvement, cardiac involvement, hand functioning, calcinosis, skin involvement, Sicca syndrome; and ii) general domains (fatigue, pain, cognition, sleep disorder). <h3>Methods</h3> A two-round international online Delphi exercise was conducted to enrich the list of instruments. Experts in lcSSc were asked to suggest additional items/outcome measures for the domains. Candidate clinician-reported outcomes (ClinROs) and performance outcomes (PerfOs) were selected during a two-day meeting utilizing a nominal group technique (NGT) comprising 3 patient research partners and 8 international experts. Patient research partners received specific-training prior to the NGT meeting to become familiar with the domains, outcomes, and research processes. <h3>Results</h3> 100 experts were invited to the Delphi exercise and 71 provided answers for at least one round. Participants who provided answers in round 1 were invited to a second Delphi exercise, rating each item on scales (range: 1-9) for feasibility, face validity, content validity, and overall appropriateness for the CRISTAL Index. 59/71 participants provided answers to at least one survey. Items endorsed by more than 50% of the experts were included for discussion at the 2 day-NGT discussion. During the NGT meeting, for each lcSSc specific domain, the list of items selected during Delphi and their characteristics were presented and each voting member was asked to discuss the relevance of these items and to propose new items if important ones were considered missing. Voting members then ranked items from "most appropriate for the CRISTAL index" to "less appropriate". At the end of each ranking, voting members were asked if they agreed with the ranking; 80% agreement was required to move to the next domain. Across the 10 lcSSc specific domains discussed, a total of 19 items (17 ClinROs and 2 PerfOs) were identified and retained as draft items for testing in the planned observational longitudinal cohort study (Table 1). <h3>Conclusion</h3> These initial results from the CRISTAL initiative are essential first steps towards the development of a composite index for lcSSc. The next steps will include selection of patient-reported outcomes (PROs) from the PROMIS item bank, existing data in the reported literature, and cognitive debriefing with patients, followed by a prospective cohort study to assess and validate the draft CRISTAL index. <h3>References</h3> [1]Lescoat A et al. Symptom experience of limited cutaneous systemic sclerosis from the Patients' perspective: A qualitative study. Semin Arthritis Rheum. 2022 Feb;52:151926. [2]Lescoat A et al. Domains and outcome measures for the assessment of limited cutaneous systemic sclerosis: an international collaborative scoping review. Rheumatology (Oxford). 2022 Aug 3;61(8):3132-3148. <h3>Acknowledgements</h3> This international project was supported by the World Scleroderma Foundation, Scleroderma &amp;. Raynaud's UK (SRUK), the Scleroderma Clinical Trial Consortium, and the US National Scleroderma Foundation. The authors thank all CRISTAL collaborators. <h3>Disclosure of Interests</h3> Alain LESCOAT: None declared, Yen Chen: None declared, Susan L Murphy: None declared, Lauryn Carroll: None declared, Nadia Vann: None declared, Paul Didio: None declared, Sarah Iya: None declared, Tiffany Phanhdone: None declared, Sue Farrington Speakers bureau: Boehringer Ingelheim, Galápagos, &amp; Janssen, Yannick Allanore Consultant of: AbbVie, AstraZeneca, Bayer, Boehringer-Ingelheim, Mylan, Janssen, Medsenic, Prometheus, Sanofi, Roche, Grant/research support from: Alpine Immunosciences, Medsenic, OSE Immunotherapeutics, David Cella: None declared, Lorinda Chung Consultant of: Boerhinger Ingelheim, Grant/research support from: Mitsubishi Tanabe, Eicos Sciences, Genentech, Kyverna, and Jannsen., Philip Clements: None declared, Francesco Del Galdo Speakers bureau: Boehringer Ingelheim, Jannsen, AstraZeneca, Consultant of: AstraZeneca, Boehringer Ingelheim, Capella, Chemomab, Jannsen, Mitsubishi-Tanabe, Grant/research support from: Abbvie, AstraZeneca, Boehringer Ingelheim, Capella, Chemomab, Kymab, Jannsen, Mitsubishi-Tanabe, Christopher P Denton: None declared, Oliver Distler Speakers bureau: 4P-Pharma, Abbvie, Acceleron, Alcimed, Altavant, Amgen, AnaMar, Arxx, AstraZeneca, Baecon, Blade, Bayer, Boehringer Ingelheim, Corbus, CSL Behring, Galderma, Galapagos, Glenmark, Gossamer, iQvia, Horizon, Inventiva, Janssen, Kymera, Lupin, Medscape, Merck, Miltenyi Biotec, Mitsubishi Tanabe, Novartis, Prometheus, Redxpharma, Roivant, Sanofi and Topadur, Consultant of: 4P-Pharma, Abbvie, Acceleron, Alcimed, Altavant, Amgen, AnaMar, Arxx, AstraZeneca, Baecon, Blade, Bayer, Boehringer Ingelheim, Corbus, CSL Behring, Galderma, Galapagos, Glenmark, Gossamer, iQvia, Horizon, Inventiva, Janssen, Kymera, Lupin, Medscape, Merck, Miltenyi Biotec, Mitsubishi Tanabe, Novartis, Prometheus, Redxpharma, Roivant, Sanofi and Topadur, Grant/research support from: 4P-Pharma, Abbvie, Acceleron, Alcimed, Altavant, Amgen, AnaMar, Arxx, AstraZeneca, Baecon, Blade, Bayer, Boehringer Ingelheim, Corbus, CSL Behring, Galderma, Galapagos, Glenmark, Gossamer, iQvia, Horizon, Inventiva, Janssen, Kymera, Lupin, Medscape, Merck, Miltenyi Biotec, Mitsubishi Tanabe, Novartis, Prometheus, Redxpharma, Roivant, Sanofi and Topadur, Monqiue Hinchcliff: None declared, Mike Hughes: None declared, Laura Hummers Consultant of: CSL Behring, Boehringer Ingelheim, Abbvie, Ortho-Dermatologics, Grant/research support from: Boehringer Ingelheim, Cumberland Pharmaceuticals, Mitsubishi Tanabe, Horizon Pharmaceuticals, Prometheus Biosciences, John Pauling Speakers bureau: Janssen, Astra Zeneca, Permeatus Inc, Sohournix Pharma, Isomab and Boehringer Ingelheim, Consultant of: Janssen, Astra Zeneca, Permeatus Inc, Sohournix Pharma, Isomab and Boehringer Ingelheim, Janet Pope: None declared, Viginia Steen: None declared, John Varga: None declared, [Peter Merkel]: None declared, Maya H Buch: None declared, Dinesh Khanna Shareholder of: Eicos Sciences, Inc, Consultant of: Acceleron, Amgen, Boehringer Ingelheim, CSL. Behring, Chemomab, Genentech/Roche, Horizon, Mitsubishi. Tanabe Pharma, Prometheus, Talaris and has received grants. from Bayer, BMS, Horizon and Pfizer.